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The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
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The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.
Maintaining America’s momentum demands that policymakers resist policies that undermine research and development incentives.
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Read our takes on the biggest stories happening in the industry.
Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.
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Sanofi Ventures, which now has $1.4 billion in total assets, will focus its investment efforts on early players working in immunology, rare diseases, neurology and vaccines.
If approved, uniQure’s gene therapy AMT-130—which slowed disease progression by 75%—would be the first genetic treatment for Huntington’s disease. A BLA submission is planned for the first quarter of 2026.
The issues the regulator found include the failure to comprehensively review complaints and product defects.
Lilly expects to open 615 high-wage roles in Texas in connection with its new facility, plus around 4,000 construction roles.
The regulatory action marks the second rejection for a spinal muscular atrophy therapy this week after Scholar Rock’s apitegromab was issued a complete response letter on Tuesday, similarly on manufacturing grounds.
The FDA is hoping to repurpose GSK’s Wellcovorin for cerebral folate deficiency; Pfizer acquired fast-moving weight-loss startup Metsera for nearly $5 billion after suffering a hat trick of R&D failures; psychedelics are primed for M&A action and Eli Lilly may be next in line; RFK Jr.’s revamped CDC advisory committee met last week with confounding results; and Stealth secured its Barth approval.
While last week’s recommended changes by CDC advisors to the MMRV vaccine schedule are unlikely to have a tangible effect on Merck’s business, the company said the removal of choice for healthcare providers is “concerning.”
In its rejection letter, the FDA flagged problems at a third-party fill-finish site owned by Novo Nordisk. Issues at this site have previously been investigated by the regulator.
The agency also pointed to the use of Tylenol and other acetaminophen products during pregnancy as being potentially linked to neurological and developmental defects in children, following a press conference Monday in which President Donald Trump did the same.
An advisory committee last year found that Zynquista’s benefit-risk profile in type 1 diabetes was unfavorable due to cases of diabetic ketoacidosis.